BackgroundBAY 81-8973 is a new full-length human recombinant factor VIII product manufactured with technologies to improve consistency in glycosylation and expression to optimize clinical performance.ObjectivesTo demonstrate superiority of prophylaxis vs. on-demand therapy with BAY 81-8973 in patients with severe hemophilia A.Patients/MethodsIn this multinational, randomized, open-label crossover study (LEOPOLD II; ClinicalTrials.gov identifier: NCT01233258), males aged 12–65 years with severe hemophilia A were randomized to twice-weekly prophylaxis (20–30 IU kg−1), 3-times-weekly prophylaxis (30–40 IU kg−1), or on-demand treatment with BAY 81-8973. Potency labeling for BAY 81-8973 was based on the chromogenic substrate assay or adjusted to the one-stage assay. Primary efficacy endpoint was annualized number of all bleeds (ABR). Adverse events (AEs) and immunogenicity were also assessed.ResultsEighty patients (on demand, n = 21; twice-weekly prophylaxis, n = 28; 3-times-weekly prophylaxis, n = 31) were treated and analyzed. Mean ± SD ABR was significantly lower with prophylaxis (twice-weekly, 5.7 ± 7.2; 3-times-weekly, 4.3 ± 6.5; combined, 4.9 ± 6.8) vs. on-demand treatment (57.7 ± 24.6; P < 0.0001, anova). Median ABR was reduced by 97% with prophylaxis (twice-weekly, 4.0; 3-times-weekly, 2.0; combined, 2.0) vs. on-demand treatment (60.0). Median ABR was higher with twice-weekly vs. 3-times-weekly prophylaxis during the first 6-month treatment period (4.1 vs. 2.0) but was comparable in the second 6-month period (1.1 vs. 2.0). Few patients reported treatment-related AEs (4%); no treatment-related serious AEs or inhibitors were reported.ConclusionsTwice-weekly or 3-times-weekly prophylaxis with BAY 81-8973 reduced median ABR by 97% compared with on-demand therapy, confirming the superiority of prophylaxis. Treatment with BAY 81-8973 was well tolerated.

In children with CMV abdominal pain, eyelid swelling, skin rash, fatigue and thrombocytopenia were more common. In children with EBV values of transaminases declined significantly faster.

The omission of standards for renal length in infants younger than 1 year may result in a statistically significant increase in the frequency of "spurious" nephromegaly. Nonetheless, there are only a few reports specifically dealing with normal kidney dimensions in infants. Based on sonographic assessments performed on a sample of 992 healthy infants, between January 2002 and December 2004, this paper sets up standards for normal kidney dimensions in children aged 0-3 months, 3-6 months, 6-9 months, and 9-12 months and establishes correlations between kidney dimensions (length, width, and volume) and body length and weight. Linear as well as non-linear nomograms, with percentiles for all the kidney variables examined, based on body length, are provided. Also, statistically significant differences in mean values (P=0.000) for all the observed neonatal kidney parameters, depending on gestational age at birth, are demonstrated. Principal advantages of our nomograms are that they are based on a large number of examined healthy infants and that kidney dimensions are related to body length. In addition, subjects are divided into four sub-annual age groups demonstrating gender-related differences in renal growth dynamics. Our linear nomograms are easier to use for routine clinical practice, but the percentile-based non-linear nomograms we present cover a much wider range of variations in normal infant kidney dimensions.

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Generalized convulsions in the afebrile infant represent a serious and etiopathogenically very heterogeneous problem. Extremely rare, as in the case of our patient, it may be due to severe hypocalcemia caused by a deficiency of vitamin D.